FDA Issues Draft Guidance on the Qualification of Drug Development Tools

On October 25, 2010, FDA issued a draft guidance entitled “Qualification Process for Drug Development Tools.” This draft guidance describes a new qualification process for drug development tools (“DDTs”), which include, among other things, biomarkers and patient reported outcome instruments. The qualification of a DDT will ensure that it can be used reliably in multiple drug development programs, rather than in just one drug-specific program. A qualified DDT will be publicly disclosed, and is therefore particularly suited to consortia and other collaborative efforts to study the use of DDTs that can be made available for use by all drug developers. FDA expects that DDTs will help speed up, not only the drug development process, but also FDA review, because FDA may apply the DDT for the qualified use without the need to reconfirm its utility in each new application. The Center for Drug Evaluation and Research (“CDER”) is currently involved in several initiatives to develop DDTs and this draft guidance furthers those efforts. These programs arise from FDA’s “Critical Path Initiative,” an ongoing effort to identify ways to enhance and improve the efficiency of the drug development process.

The draft guidance lays out a two-stage process for a qualification determination. Stage 1, the consultation and advice stage, begins with a DDT sponsor submitting a letter of intent (“LOI”) requesting that CDER evaluate the potential use of particular DDT. If CDER grants the request, the Division will ask for a more detailed briefing package. In addition, the Agency will form a Qualification Review Team (“QRT”) to provide ongoing advice to the submitter, beginning with an initial meeting to discuss the DDT, including its use, supportive data, and any additional data necessary for qualification. After the submitter has collected the recommended data, CDER will review the data and determine whether the DDT is ready for review.

In Stage 2, the qualification review stage, the submitter submits a qualification package to CDER that contains analyses of any supporting studies. CDER will then evaluate the information submitted, and may hold a public discussion as necessary. Finally, CDER will issue a Statement of Qualification if it determines that the DDT is “qualified.” The determination will be issued as a draft guidance and posted on FDA’s website for comment. Once the Agency evaluates the comments, it will publish the final guidance in the same manner.

Although this draft guidance is an important first step in establishing a regulatory framework to qualify biomarkers and other DDTs, several limitations are noteworthy. First, the draft guidance only describes the procedure of how CDER will qualify a DDT; it does not address the level of evidence needed to qualify it. Second, the procedures outlined do not apply to a DDT developed for a company’s propriety use. Instead, FDA suggests that information on such DDTs should be submitted to the IND, NDA, or BLA. Third, the draft guidance does not contain any timelines as to when CDER would respond to an LOI, make a qualification decision, or schedule meetings with the DDT sponsors. Industry may want to consider commenting on these “gaps” in their comments to the docket.

Comments to the draft guidance must be submitted by January 24, 2011 in order to ensure consideration.